There is a puzzle when it comes to the controversial idea of using cord blood for autism, because two of the strongest proponents are the autism cord blood program at Duke and for-profit, unproven stem cell clinics. On first glance it seemed like this was a bit of an odd couple to me.
The links between the Duke autism program and one particular Panamanian stem cell clinic called generically enough “The Stem Cell Institute” appear to go beyond just their enthusiasm about cord blood for autism.
Those apparent connections involve a mega-donor who has given a vast sum perhaps as high as $10s of millions to Duke’s autism cord blood group. Furthermore, The Stem Cell Institute says the donor and people in his circle have also been their customers. The clinic also makes another particularly startling claim I go into later in the past.
What’s going on in this space?
Cord blood must work for autism?
By way of brief background, what does the published science tell us about the hypothesis of using cord blood for autism?
Duke’s own recently-published clinical trial is one of the strongest in design of any yet in this area. In that trial, cord blood clearly did not help autism, but puzzlingly after their paper came out the Duke group said they were “encouraged” by it. This assessment seems based on a post-hoc analysis that is somewhat questionable in my view
Remarkably the Duke team has subsequently begun not one but two more new, related trials.
How can they still be so enthusiastic?
I don’t know.
There always seems to be some rationalization to keep them going.
Things like this pop up, “If only we had designed our trial differently and excluded some patients, we would have seen benefit”, and so on. Maybe now they think that instead of cord blood it’ll be better to use lab-grown cord stem cells?
It feels to me like they just can’t let go of their idea that cord cells must help autism despite the data generally saying otherwise so far both from their own and other trials.
Other clinical trial work also shows no benefit
In terms of other cord blood autism trials, a Sutter trial run right here out of Sacramento also found no benefit. From the Sutter trial paper:
“There were also no statistically significant differences between scores on the two primary or secondary endpoints after infusion with AUCB versus infusion of placebo.”
Note that AUCB stands for autologous umbilical cord blood.
Despite the negative clinical trial data, upbeat statements about cord blood for autism persist and more children are being enrolled in trials. Is there anything there but wishful thinking? It’s hard to say, but I’m skeptical.
For-profit stem cell clinics and autism
More generally, the research area of stem cells and cord blood for autism has been complicated by the numerous stem cell clinics who seem to be trying to make money that way. These firms include some clinics in the U.S. and in other countries such as that Panamanian one The Stem Cell Institute, run by Neil Riordan. They sell unproven stem cell offerings of various kinds for autism. Some make unsubstantiated claims about positive effects of stem cells for autistic kids.
Riordan’s clinic specifically infuses autistic kids with cord stem cells amplified in a lab, hoping for benefit. They market this and other cellular offerings for many conditions including some adult health issues, but I tend to think of this business as most well-known for its autism offerings. You can see a past screenshot from their website below as to the diverse menu of what they have been marketing.
Why would stem cells, often the same product, help so many diverse conditions?
The for-profit stem cell clinics and academic or large hospital autism/cell therapy researchers like Duke’s don’t operate in entirely separate bubbles, of course. The stem cell world is interconnected in what I’ve called one large stem cell ecosystem.
A long-standing concern has been the possibility that the portrayal of the autism clinical trial efforts by Duke, Sutter, and potentially others could be used by clinics like the Panama one to more effectively market their unproven offerings. Note that the Panama clinic has been quite successful at PR to attract customers, including appearing on the Joe Rogan show.
Flipping things around, another potentially troubling question comes to mind. Could the clinics also help the Duke autism team or other academics in some ways too that are not necessarily in the best interest of the children?
Duke-Panama stem cell clinic connection?
As I’ve tried to learn more about all of this over the years, I found that the Panama stem cell clinic often kept popping up in a parallel way to the Duke Autism Program on the web with different searches. Some patients mention both.
More concretely, Duke has reportedly received more than USD $10 million (media reports range from $15 million to $26 million or even $40 million) for its autism research from a philanthropic organization called the Marcus Foundation, which also has connections to The Stem Cell Institute clinic in Panama, at least according to the clinic. The Marcus Foundation is led by Home Depot founder and billionaire, Bernie Marcus.
It turns out at least according to Riordan that Marcus apparently himself got unproven stem cells at The Stem Cell Institute and maybe one or more of his family or friends possibly went there too. From Riordan:
“Bernie Marcus founded and chairs The Marcus Foundation, whose focus on children’s health, medical research, free enterprise, and Jewish causes has converged with his support for stem cell research. “It’s done such amazing things for me that I’ve become an advocate for stem cells,” he says. “My experiences in Panama have really moved me to try and get stem cells into the United States. I don’t know where it’s not going to be useful. The work in Panama is proving to be a blessing in disguise for a lot of people. It’s the opening of a new era, and especially umbilical cord stem cells.”
Such an enthusiastic quote is a kind of valuable endorsement for the clinic, right?
Panama clinic leader was Duke grant reviewer?
Why is this connection a big deal? There seems to be more to it.
Remarkably, according to Riordan in another piece he wrote responding to a somewhat critical Spectrum News article, he says that he was a reviewer of Duke’s application to get that big funding from the Marcus Foundation (emphasis mine):
“I know Dr. Kurtzberg at Duke. Bernie Marcus, who funded the Duke trials, has been a patient in Panama (public information) and saw children with autism benefitting from treatment with umbilical MSCs first hand. I was asked to review the Duke proposal for the Marcus Foundation and was at the Foundation Board meeting that led to the funding.”
If correct, to me that is quite a bombshell revelation. It seems like the implication is that he helped Duke get that huge funding.
As to his statement about knowing Kurtzberg, Riordan also has another stem cell clinic firm called the Riordan Medical Institute (RMI) and on that website there is a picture of Riordan and Kurtzberg together. Note that the screenshot also contains the original caption from RMI.
Last month, I reached out to the Marcus Foundation asking to confirm whether Mr. Marcus went to the clinic, about the foundation’s support of Duke’s autism work including their new trials, and any ties with The Stem Cell Institute such as Riordan potentially being a reviewer including for the Duke grant proposal.
So far I have not heard back so I cannot confirm Riordan’s statements on the web.
More questions on Duke autism program
This all prompted some new questions.
Could this situation change how the Duke autism researchers approach their own research and how they talk about it publicly?
Would they be less likely to be critical (either publicly or with parents of autism children) of unproven stem cell clinics that claim to treat autism via cord blood? Seven years ago, I interviewed Kurtzberg here on The Niche, see part 1 and part 2. In the second half of the interview I asked her about stem cell clinics including ones selling offerings for CP and autism. Back then, she expressed concern about such clinics.
Could they be more likely to publicly make statements that promote cord blood for autism beyond the scope of what’s proven by the data?
Also, in a sense, does the Duke autism program benefit if Riordan’s Panama clinic is more successful because they share that central passion of “core blood for autism!”?
Broader concerns including costs to families
There also have been a few indications on the web that the Duke Autism Program possibly has been requiring some patient families to pay large amounts for cord blood injections including for the families to get access off-study through compassionate use. If this is correct and the costs go well beyond the typical expenses people sometimes have to cover indirectly to be in trials (e.g. mostly just travel-related costs), then I find that kind of arrangement concerning.
It’s unusual to have mandatory patient payments just to enroll in a trial or compassionate use program. I’ve written critically before about how Northwestern’s now-defunct autoimmune hematopoietic stem cell transplant (HSCT) program required sometimes huge patient payments for enrollment in trials or off-study “treatment”. This kind of pay-for-play-like arrangement raises complex ethical issues, even if approved by the FDA.
Also, especially in light of the Duke Autism Program getting so much funding from Marcus, why would it be necessary to require payments from any families to have their children be involved?
When I searched this summer on GoFundMe it was easy to find fundraising campaigns related to cord blood or stem cells for autism that prominently mentioned needing funds for the Duke autism program or to go to Panama. Some mentioned both Duke and Panama in the same campaign.
A few campaigns suggested Duke was requiring fees of around $15K in some cases like this one. If such fees are only for compassionate use, how many families have paid to have their children get infused by Duke with something that may have no benefit and does have some risks?
I saw a real red flag in another GoFundMe campaign. There seems to be a direct logical progression from an overly upbeat interpretation of Duke’s cord blood autism results to a family deciding to spend $20K at The Stem Cell Institute in Panama (emphasis mine):
“Stem cell therapy is a groundbreaking procedure that is unfortunately not offered in the United States at this time, although Duke has started doing some clinical trials with impressive results for autistic children.
We have decided to take Noah to The Stem Cell Institute in Panama City, Panama. This procedure takes 5 days to complete with an overall delivery of 40 million umbilical stem cells given to Noah via IV. I have done hours worth of research and believe that this therapy is right for him. Every patient has had significant speech and vocabulary improvements, decreased behavioral outbursts and increased social awareness.
The total cost is $20,000…“
Every patient? Where are the impressive data?
Have statements from Duke’s team given parents a potentially inaccurate impression? If many parents have this kind of impression and make this kind of Duke-Panama clinic connection, one could see how The Stem Cell Institute might be very upbeat with the Duke autism group.
If the Duke team keeps getting millions of dollars for “stem cells for autism” is that an incentive for the Duke team to just keep going even if arguably the data say it should hit pause?
One should keep in mind that we are talking about dozens of children being enrolled by their parents in these clinical trials and/or injected off-study with experimental cells, practices that in this case are in my view not scientifically well-justified at this point and again pose risks. Note that there is now a petition, signed by more than 13,000 people, seeking to halt Duke’s autism cord blood trials.
Since for its new pediatric trial the Duke team seems to be switching to laboratory-expanded cord cells, similar to The Stem Cell Institute clinic, how does that impact potential risks to the children who are the participants in the trial? What about economic impacts on the families involved? There is definitely the possibility of false hope here too.
In my view there needs to be far more transparency here.